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Lee Rubin, PhD

Stem Cell and Regenerative Biology
Harvard Stem Cell Institute
Sherman Fairchild, Ground Floor
7 Divinity Ave.
Cambridge, Massachusetts, United States

Lee Rubin investigates the key molecular mediators of different neurodegenerative diseases, with the ultimate goal of finding effective preclinical therapeutic candidates. His research group discovered that a circulating protein, GDF11, has the ability to reverse some of the changes in the CNS associated with aging. They are actively exploring the therapeutic implications of these observations.

The Rubin group takes advantage of their ability to produce large numbers of patient-derived induced pluripotent stem (iPS) cell lines and of effective means of deriving large numbers of differentiated neurons from them. They have set up an array of techniques that allow them to identify early cellular and physiological changes in neurons as they become diseased. For example, they have identified new targets for the treatment of the motor neuron disorders –  Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS).

Dr. Rubin received his Ph.D. in Neuroscience from The Rockefeller University and completed Postdoctoral fellowships in Pharmacology from Harvard Medical School and in Neurobiology from Stanford University School of Medicine. He has a broad experience in both academia and industry, particularly in the realms of cell-based assays and drug discovery.


Representative Publications:

Genetic Modifiers Ameliorate Endocytic and Neuromuscular Defects in a Model of Spinal Muscular Atrophy

Systemic Nature of Spinal Muscular Atrophy Revealed by Studying Insurance Claims

Blocking P62-Dependent SMN Degradation Ameliorates Spinal Muscular Atrophy Disease Phenotypes

The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy


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